[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"sickling-disorder-due-to-hemoglobin-s\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:sickling-disorder-due-to-hemoglobin-s":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,52],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100594149","phase-1-131i-apamistamab-based-conditioning-for-hematopoietic-stem-cell-transplant-hsct-in-advanced-sickle-cell-disease-scd-100594149",false,"NCT07015684","131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)","Open-label, Single-Center, Phase 1 Study to Estimate the Minimum Effective Dose (MED) of 131I-apamistamab for Non-myeloablative Conditioning in Patients With Severe Sickle Cell Disease","Inclusion Criteria:\n\n* Availability of an HLA-matched sibling donor\n* Patients with sickle cell anemia (Hb SS, Sβ0 thalassemia or severe SC) who are 12 - 50 years of age inclusive AND who have 1 or more of the following:\n\n  1. Clinically significant neurologic event (stroke) or any neurological deficit lasting at least 24 hours. Stroke will be defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI or cerebral arteriopathy requiring chronic transfusion therapy.\n  2. History of two or more episodes of ACS in the 2-year period preceding enrollment despite supportive care measures (i.e. asthma therapy and\u002For hydroxyurea).\n  3. History of three or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and\u002For treatment with hydroxyurea).\n  4. Administration of regular RBC transfusion therapy, defined as receiving 8 or more transfusions per year for 1 year or more to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and ACS)\n  5. An echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity \\> or equal to 2.7 m\u002Fsec or pulmonary hypertension diagnosed by right heart catherization.\n  6. Sickle hepatopathy defined as EITHER ferritin \\>1000mcg\u002FL OR direct bilirubin \\>0.4mg\u002Fdl but \\\u003C5xULN AND platelet count \\\u003C250,000\u002FuL at baseline\n* Adequate organ functions as defined as:\n\n  1. ECOG performance status of 2 or better\n  2. Cardiac function: LVEF of 40% or greater\n  3. Pulmonary Function: Pulse oximetry with a baseline oxygen saturation of 85% or greater and corrected DLCO of 40% or greater\n  4. Hepatic Function: Serum conjugated (direct) bilirubin less than 5x upper limit of normal for age as per local laboratory, ALT and AST less than 5 x upper limit of normal as per local laboratory. Patients whose hyperbilirubinemia is the result of hyperhaemolysis, or a sever drop in hemoglobin post blood transfusion are not excluded.\n  5. Absence of liver cirrhosis, bridging fibrosis and active hepatitis as documented by liver biopsy for patients with evidence of iron overload by serum ferritin or MRI. The histological grading and scale described by Ishak and colleagues (1995) will be used.\n\nDonor Eligibility and Selection Criteria\n\n1. Donor should be evaluated for eligibility to donate by an independent physician not directly caring for the patient on study protocol.\n2. Donor is willing to sign informed consent allowing the use of the PBSC product for the HSCT of the recipient.\n3. Donor cannot be pregnant or lactating and must agree to contraception until after the donation procedure is complete.\n4. Testing negative for HIV and viral hepatitis\n5. Free of Hb S (defined as Hb S less than 50%) and other hemoglobinopathies that are symptomatic or of clinical significance.\n6. Targeted minimum stem cell dose of 5.0 x 10e6 CD34 cells\u002FKg of recipient weight\n7. Fulfills standard criteria for eligibility as a donor for HSCT.\n\nNote: HSCT can be deleterious for the developing fetus and pregnant mother due to the conditioning regimen, GVHD prophylaxis and treatment. Agents used in this study such as cyclophosphamide are pregnancy risk factor category D. Sirolimus is pregnancy risk factor category C. Radiotherapy also used (TBI) is a well-known teratogenic agent. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study and for at least 1 year post transplant.\n\nFinally, pregnancy and lactation restrictions and contraception requirements are also applicable to the donor. Filgrastim or other G-CSF analogous are pregnancy risk factor category C. The restriction lasts for 4 weeks after stem cell donation.\n\nExclusion Criteria:\n\n1. Pulmonary dysfunction defined as DLCO (corrected for hemoglobin and alveolar volume) \\\u003C 40% of predicted OR baseline oxygen saturation of \\\u003C85% or PaO2 \\\u003C70.\n2. Severe cardiac dysfunction defined as ejection fraction \\\u003C35%.\n3. Impaired renal function defined as GFR \\\u003C40.\n4. Hepatic dysfunction defined as bridging (portal to portal) fibrosis or cirrhosis of the liver OR transaminases \\>5x ULN for age.\n5. Clinical stroke within 6 months of anticipated transplant\n6. Karnofsky performance score \\\u003C 50%\n7. HIV infection\n8. Uncontrolled viral, bacterial, fungal, or protozoal infection at the time of study enrollment.\n9. Have circulating HAMA noted on initial screening.\n10. Have received prior radiation to maximally tolerated levels to any critical normal organs\n11. Patients with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate HSCT.\n12. Patients' unable to understand the nature and risks inherent in the HSCT process.\n13. History of non-compliance severe enough in the estimation of the treating team to preclude the patient from undergoing unrelated donor transplantation.\n14. Patient is pregnant or lactating.\n15. Inability to provide adequate transfusion support or increased risk immunohematological complications due presence of anti-RBC antibody against stem cell donor.\n16. Patients with any history of radiation therapy.","ALL","12 Years","50 Years",{"count":20,"type":21},24,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","The purpose of this study is to find the smallest amount of the 131 I-apamistamab needed for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant. This is the first time 131 I-apamistamab is being used for advanced Sickle Cell Disease (SCD) in the setting of allogeneic stem cell transplant. 131 I-apamistamab is an investigational product. This means that 131 I-apamistamab has not been approved by the Food and Drug Administration (FDA) for medical use in patients.\n\nThe study treatment that is given before the transplant is called the conditioning regimen. In this study, the investigators are adding a drug called 131 I-apamistamab instead of the conditioning regimen typically given before a stem cell transplant.",[27],"Sickling Disorder Due to Hemoglobin S",[29,30,31,32,33,34,35,36,37,38],"severe sickle cell disease","severe SCD","sickle cell disease","sickle cell","advanced SCD","advanced sickle cell disease","anemia","sickle cell disorders","hemoglobin S (HbS) Disease","Hemoglobin S Disease","RECRUITING","2026-06-08",{"date":42,"type":43},"2026-06-10","ACTUAL",{"date":45,"type":43},"2025-04-28",{"date":47,"type":21},"2032-03-12",{"name":49,"class":50},"Columbia University","OTHER",1,{"id":53,"slug":54,"hasResults":11,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":4,"eligibilityCriteria":58,"healthyVolunteers":11,"sex":16,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":4,"studyType":22,"phases":63,"briefSummary":65,"conditions":66,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":80},"100377950","phase-2-a-trial-to-assess-haploidentical-t-depleted-stem-cell-transplantation-in-patients-with-scd-100377950","NCT04201210","A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD","A Phase II Stratified Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With Sickle Cell Disease With no Available Sibling Donor","Inclusion Criteria:\n\n* Age 2yrs to 35yrs\n* Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0\u002F+\n* Study specific consent given\n* Preexisting severe or moderate SCD related complications:\n\n  * Clinically significant neurological event (stroke) or deficit\n  * Silent crisis, neurocognitive deficit\n  * Pathological angio-MRI with TOF Sequence\n  * TCD velocity \\>200 cm\u002Fs at 2 occasions \\>1 month apart\n  * More than 5 vaso-occlusive crises (VOC) in the past 1 year or more than 20 VOC in a lifetime\n  * Two or more episodes of acute chest syndrome (ACS) in a lifetime or one episode of ACS in the past 24 months\n  * Chronic transfusion requirement or more than 8 transfusions or one exchange transfusion in a lifetime\n  * Transfusion-refractory allo-immunization\n  * More than five SCD-related hospitalizations in a lifetime\n  * Beginning pulmonary hypertension\n  * Osteonecrosis at more than 2 sites\n  * Beginning SCD Nephropathy\n  * Recurrent priapism (\\>2)\n\nExclusion Criteria:\n\n* Karnofsky or Lansky Performance Score \\\u003C 70%\n* Patients with donor-specific antibodies (DSA) against the potential stem cell donor by either\n\n  * Cell-based crossmatched assays (Complement-dependent cytotoxicity; CDC) or\n  * Flow cytometry crossmatch test or\n  * Solid-phase immunoassays (SPI) or\n  * Modified SPI such as C4d and C1q assays Whichever method the participating center is experienced in.\n* Patients with major AB0 incompatibility defined according to EBMT Handbook, Edition 2019 Tab 23.1.:\n\nABO incompatibility Recipient Donor Major O A O B O AB A AB B AB\n\n* Cardiac function:\n\n  * Ejection fraction at rest \\\u003C45.0% on echocardiography or\n  * Shortening fraction of \\\u003C27.0% by echocardiogram or radionuclide scan (MUGA)\n  * Patients with \\> grade II hypertension by Common Toxicity Criteria (CTC)\n* Renal function:\n\n  * Estimated creatinine clearance (for patients \\> 12 years) greater than 50.0 mL\u002Fminute\n  * for pediatric patients (\\> 1 year to 12 years), GFR estimated by the updated Schwartz formula ≥ 90.0 mL\u002Fmin\u002F1.73 m2. If \\\u003C 90 mL\u002Fmin\u002F1.73 m2, renal function must be measured by 24-hour creatinine clearance or nuclear GFR and must be \\> 70.0 mL\u002Fmin\u002F1.73 m2 or\n  * Creatinine clearance below threshold defined for stem cell transplantation according to local clinical standard\n* Pulmonary function:\n\n  * DLCO \\>50% (adjusted for hemoglobin), and FVC and FEV1≥50%; children unable to perform for PFTs, O2 saturation \\\u003C92% on room air.\n* Liver function:\n\n  * Total bilirubin \\> 2x the upper limit of normal (unless elevated bilirubin is attributed to Gilbert's Syndrome) and ALT\u002FAST \\> 2.5x the upper limit of normal.\n  * Chronic active viral hepatitis\n* Women who are pregnant (positive serum or urine βHCG) or breastfeeding. Note: Women of childbearing potential must have a negative serum pregnancy test at study entry.\n* Adults of reproductive potential not willing to use an effective method of birth control during study treatment and for at least 12 months thereafter,\n* History of uncontrolled autoimmune disease or on active treatment\n* Patient unable to comply with the treatment protocol\n* Prior autologous or allogeneic hematopoietic stem cell transplant\n* Vaccination with a live virus vaccine during the trial\n* HIV infection\n* Patients with a history of psychiatric illness or a condition which could interfere with their ability to understand the requirements of the study (this includes alcoholism\u002Fdrug addiction)\n* Patients unwilling or unable to comply with the protocol or unable to give informed consent.\n* Concurrent severe or uncontrolled medical disease (e.g. uncontrolled diabetes, congestive heart failure, myocardial infarction within 6 months prior to the study, unstable and uncontrolled hypertension, chronic renal disease, or active uncontrolled infection) which by assessment of the treating physician could compromise participation in the study","2 Years","35 Years",{"count":62,"type":21},212,[64],"PHASE2","HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß\u002FCD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.",[67,38,68,69,27,70],"HbS Disease","Sickle Cell Anemia","Sickle Cell Disorders","Sickle Cell Disease","2024-05-10",{"date":73,"type":43},"2024-05-13",{"date":75,"type":43},"2021-06-30",{"date":77,"type":21},"2030-03-31",{"name":79,"class":50},"University of Regensburg",9]